Therapeutic Approaches to Genetic Disease
Dr. Conklin uses induced pluripotent stem (iPS) cells, both from patients and engineered to have particular mutations to model human disease. His lab is developing new genome engineering methods in human iPS cells to identify therapeutic targets in cardiac, motor neuron and retinal diseases. Trainees will utilize CRISRP technology for therapeutic genome editing, then test the effect of genome editing in diseases modeled in iPS cells. The combination of human iPS cells and genome editing provide unprecedented opportunities to explore new areas of biology and discover new therapies for disease. Dr. Conklin has mentored many graduate students and postdoctoral fellows in the lab. He has on-going collaborations with Dr. Shen that has led to a publication in Nat Genetics 2019 and Dr. Lakkaraju studying water transport across the retinal pigment epithelial (RPE), as a functional measure of gene correction in Best’s disease. Scholars interested in the use of state-of-the-art therapeutic genome editing strategies and stem cell biology will find opportunities in Dr. Conklin’s lab.
To Learn More:
Gene Research, Gene Therapy, Stem Cell Research
Learn more about UCSF Ophthalmology faculty research.